Where hereditary angioedema treatment is heading
Hereditary angioedema · Updated Nov 14, 2026
Hereditary angioedema (HAE) is a rare inherited condition, affecting about 1 in 50,000 people (roughly 6,000-7,000 Americans), that causes sudden, painful swelling of the skin, gut and airway; throat swelling can be fatal. Most cases come from too little or faulty C1 inhibitor, leading to excess bradykinin. Over the past decade, preventive injections and pills have made attacks rare for many, and 2025-2026 brought new approvals and the first gene-editing results that could make HAE a one-time treatment.
- 33studies open or running
- 13phase 3 trials
- 260papers published this year
- 2NIH-funded projects
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Now
Nov 2026
One-time gene editing worksHappened 87% fewer attacks
Lonvo-z cut attacks 87% vs placebo; over 60% were attack-free.
87%: fewer attacks than with placebo.
Less frequent preventionHappened 81% fewer attacks
Donidalorsen every 4 weeks cut attacks by 81%; approved 2025.
81%: fewer attacks with 4-weekly dosing.
A pill for attacksHappened 1.61 hours to relief
Sebetralstat brought relief in a median 1.61 hours vs 6.72 with placebo; approved 2025.
1.61: hours median to symptom relief, vs 6.72 with placebo.
A daily pill for preventionHappened 83% fewer attacks
Deucrictibant XR cut attacks by 83% in phase 3.
83%: fewer monthly attacks than with placebo.
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6 months
by May 2027
A twice-yearly injectionLikely
Astria's navenibart ALPHA-ORBIT (157 people) finishes December 2026.
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12 months
by Nov 2027
FDA review of gene editingPossible
Intellia began a rolling FDA application for lonvo-z; a decision could come in 2027.
Deucrictibant filingPossible
Pharvaris is expected to seek approval for once-daily deucrictibant XR.
-
3 years
by late 2029
An siRNA given rarelyPossible
ADARx's ADX-324 STOP-HAE (90 people) is due November 2027.
Treatment for childrenPossible
Donidalorsen and other drugs are being tested in children; due 2029.
-
5 years
by late 2031
Gene therapy to restore C1 inhibitorLong shot
BioMarin's BMN 331 aims to make the body produce working C1 inhibitor; due November 2028.
The research paths
Gene editing and gene therapy
87% fewer attacksDetails and 1 trialsHide
One-time treatments that may end the need for regular drugs.
WatchLonvo-z FDA review; BMN 331.
Less frequent prevention
81% fewer attacksDetails and 4 trialsHide
Antisense, siRNA and long-acting antibodies given monthly to twice yearly.
WatchALPHA-ORBIT; STOP-HAE.
- Navenibart, a twice-yearly injection (ALPHA-ORBIT)Astria Therapeutics · 157 people · fully enrolled Dec 2026
- ADX-324, an siRNA (STOP-HAE)ADARx Pharmaceuticals · 90 people · enrolling Nov 2027
- Donidalorsen in childrenIonis · 20 people · enrolling Jun 2029
- Plasma-derived C1 inhibitorOctapharma · 124 people · enrolling Dec 2026
Oral drugs
83% fewer attacksDetails and 2 trialsHide
Pills for both attacks and prevention.
WatchDeucrictibant filing.
Borrowed from other conditions
From Sickle cell disease and ATTR amyloidosis research
CRISPR gene editing
Phase 3
The same in-body editing approach also tested for ATTR amyloidosis.
From Acquired angioedema research
Deucrictibant
Phase 3
Also tested for angioedema from acquired C1-inhibitor deficiency.
Open to join now
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HAE
ADX-324
Locations and eligibility → -
HAE
Deucrictibant
Locations and eligibility → -
Children with HAE
Donidalorsen
Locations and eligibility → -
HAE
Plasma-derived C1 inhibitor
Locations and eligibility →
See every recruiting HAE trial →
When the trials finish
Completion dates the trials registered. Results usually follow 3 to 12 months later.
- Heating up
- Maturing
- Early
- Cooling
- Fully enrolled, waiting on results
- Still enrolling
What just happened
Daily deucrictibant pill cuts attacks 83%
CHAPTER-3.
CRISPR treatment cuts attacks 87% in phase 3
HAELO, NEJM.
Standard treatment today
- Treating attacksC1 inhibitor, icatibant, ecallantide or sebetralstat (oral).
- PreventionLanadelumab, berotralstat, garadacimab, donidalorsen or C1 inhibitor.
- Before proceduresShort-term C1 inhibitor.
- Children and pregnancyPlasma-derived C1 inhibitor is preferred; pediatric trials ongoing.
How to read this page, and sourcesHow to read this page, and sources
- HAE is different from common allergic swelling and does not respond to antihistamines or epinephrine.
- Forecasts are editorial judgments by Find a Trial, not predictions from sponsors or regulators.
See HAE trials recruiting now →